New Drug APG-2575 Granted Orphan Drug Designation for the Treatment of Chronic Lymphocytic Leukemia

Last updated: November 2020

The U.S. Food and Drug Administration (FDA) has granted new drug APG-2575 orphan drug designation to treat chronic lymphocytic leukemia (CLL). APG-2575 is a novel, orally administered drug that is being developed by Ascentage Pharma, a biotechnology company that develops drugs for cancers, hepatitis, and other conditions.1

APG-2575 was granted Orphan Drug Designation (ODD) for CLL. APG-2575 has another ODD for the treatment of Waldenström Macroglobulinemia (WM). WM is a rare blood cell cancer. This new ODD for APG-2575 is for CLL.1

What is an orphan drug resignation?

Orphan drugs prevent, diagnose, or treat a rare disease or condition. A rare disease or condition affects less than 200,000 people in the United States. The U.S. government provides incentives and support for companies to develop orphan drugs. This support encourages companies to develop medicines for rare diseases or conditions.1

How does APG-2575 work?

APG-2575 is a type of drug called a Bcl-2 inhibitor. It works to treat various blood cancers. APG-2575 blocks a gene called Bcl-2 and restores apoptosis (cell death) in cancer cells.1

Why is this a necessary approval for chronic lymphocytic leukemia?

Chronic lymphocytic leukemia is a type of leukemia (blood cancer).

There is a significant need for medicines that help people with CLL survive longer. Research shows:1

  • There will be about 21,040 new cases of CLL in the United States this year
  • There will be about 4,060 deaths from CLL in the US this year

Less than 200,000 people in the United States have CLL. This number makes APG-2575 a candidate for orphan drug designation. Medicines called Bruton tyrosine kinase (BTK) inhibitors and Bcl-2 inhibitors have helped people with CLL. However, there is a need for medicine that is safe and effective for treating CLL.1

Evidence for APG-2575

APG-2575 is one of a few Bcl-2 inhibitors in development worldwide. It is the first Bcl-2 inhibitor both developed and in clinical trials in China. APG-2575 will enter Phase Ib and II clinical studies in China, Australia, and the United States. It will be studied alone and combined with other medicines in people with relapsed or refractory CLL or small lymphocytic lymphoma (SLL). The study is now looking for people to join in the United States and Australia.1

What are the possible side effects of APG-2575?

Information about side effects is not yet available. Further studies will provide more details about the side effects of APG-2575.

Things to know about APG-2575

APG-2575 is still in testing. Further clinical trials will tell us more about how safe and effective the drug is for people with CLL. We will also learn more about side effects, effects in pregnancy, and drug interactions.1

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